SOURCE: Pipex Pharmaceuticals, Inc.

November 26, 2007 07:30 ET

Pipex Pharmaceuticals Announces CRADA for Huntington's Disease Development Program With Anti-Copper COPREXA

Study Evaluates Potential Disease Modifying Properties of COPREXA in Another Deadly CNS Disease in Which Copper Is Implicated

ANN ARBOR, MI--(Marketwire - November 26, 2007) - Pipex Pharmaceuticals, Inc. (AMEX: PP), a specialty pharmaceutical company developing innovative late-stage drug candidates for the treatment of neurologic and fibrotic diseases, announced that it has entered into a Cooperative Research and Development Agreement (CRADA) with the Veterans Affairs (VA) Medical Center in Ann Arbor, MI to evaluate COPREXA (oral tetrathiomolybdate), its lead anti-copper drug candidate, in preclinical studies as a treatment for Huntington's Disease (HD), a life threatening and debilitating neurodegenerative disease.

Steve H. Kanzer, Chairman & Chief Executive Officer of Pipex, said, "Given the selective nature of COPREXA to reduce free copper levels and its recently demonstrated ability to reduce insoluble amyloid-beta by 40% (p < 0.05) in a murine Alzheimer's disease model, coupled with our recent results demonstrating elevated levels of 'free' copper in the serum of Alzheimer's disease patients (p < 0.02), this CRADA underscores our commitment to exploring the potential role that elevated CNS copper may play in other serious life threatening diseases in which elevated CNS copper is suspected, such as HD. We look forward to sharing the results of these studies with the Huntington's scientific community as soon as results are available."

Mr. Kanzer continued, "Currently, there are no adequate or disease modifying treatments for Huntington's disease and this research program may represent an important step forward in the understanding of the disease and may be a potential disease modifying approach for the 70,000 patients worldwide currently suffering from this deadly disease."

Dr. Roger L. Albin, Professor of Neurology at the University of Michigan and lead investigator on this CRADA at the VA Medical Center in Ann Arbor, MI, stated, "The scientific rationale for evaluating COPREXA is strong and it is exciting to evaluate a compound which we know is tolerated well in patients with neurodegenerative disease."

Market Opportunity for COPREXA in Huntington's Disease

Huntington's disease is a genetic neurodegenerative disease characterized by movement disorder, dementia and psychiatric disturbance. Early symptoms might affect cognitive ability or mobility and include depression, mood swings, forgetfulness, clumsiness, involuntary twitching and lack of coordination. Later, concentration and short-tem memory diminish, and involuntary movements of the head, trunk and limbs increase. Eventually, the person is unable to care for himself or herself. Death follows from complications including choking, infection or heart failure.

HD has been diagnosed in approximately 30,000 patients in the U.S. and approximately 40,000 in Europe. Patients with late stage disease require continuous nursing care, often in nursing homes, with an estimated annual cost to the U.S. economy of up to $2.5 billion. Presently, there is no effective approved treatment or cure for HD.

For further information on the disease, please visit the Huntington's Disease Society of America at

Huntington's Disease Model, R6/2 Trangenic Mouse Model

HD is caused by an expansion of a CAG repeat in exon 1 of the huntingtin gene, which encodes a protein suggested to be associated with synaptic vesicles and microtubules in neurons. There is evidence that huntingtin directly and/or indirectly plays a role in vesicle trafficking and neurotransmission.

The R6/2 mouse expresses exon 1 of the human HD gene, containing 150 CAG repeats. Consequently, the mice develop neurological symptoms that resemble many of those seen in HD. These include deficits of motor coordination, altered locomotor activity, impaired cognitive performance and seizures. Specific behavioral tests detect cognitive symptoms around week 3-4, whereas motor symptoms typically develop later. In most breeding colonies, the R6/2 mice die at week 12-14 for unknown reasons. At this stage, in the brain there is significant atrophy of the striatum and neocortex and widespread occurrence of neuronal intranuclear inclusions of the mutant huntingtin protein.

Recent scientific studies suggest a role for copper in the pathogenesis of neurodegenerative diseases, including HD. This research describes an increased copper deposition both in HD post-mortem striatum and in cortex and striatum of murine genetic models of HD, including R6/2 mice. As seen in Wilson's disease, copper is definitely toxic to striatal and other basal ganglia neurons, producing various motor and behavioral problems. Copper could also affect aggregation of mutant huntingtin protein. Based on available data, increased neuronal copper seems to be an important potentiator of neurodegeneration, making COPREXA a rational therapeutic treatment modality for the treatment of HD.

COPREXA's specificity and unique mechanism of action for rapidly lowering toxic levels of free copper in the CNS, combined with its history of success in completed pivotal clinical trials of neurologically-presenting Wilson's disease, may make it an excellent candidate to evaluate in human clinical trials for HD, a movement disorder that, like Wilson's disease, affects the basal ganglia. This hypothesis is further supported by findings since 1991 that, following their death, HD patients had 67% more copper in an area of the brain known as the putamen as well as elevated levels in their substantia nigra compared to persons without HD. Dexter DT et al., Brain 114 (Pt 4):1953-75 (1991) Pubmed ID: 1832073. The conduct of any such trials, as well as the design and conduct of necessary registration trials sufficient to support a potential NDA or Supplemental NDA of COPREXA for HD, are subject to FDA authorization and, if such trials are successful, FDA approval to market COPREXA for this indication. COPREXA's ability to specifically bind and lower toxic free copper levels differentiates it from other non-specific metal chelating compounds previously investigated for neurodegenerative diseases.


COPREXA is an oral, small-molecule, anti-copper agent that is highly specific for the reduction of free copper in serum, the most toxic form of copper in the body, and is thus ideally suited for the treatment of central nervous system (CNS) diseases in which abnormal serum and CNS copper homeostasis are implicated. We are also developing COPREXA™ for fibrotic disorders based upon the rationale that the fibrotic disease process is dependent upon the availability of free copper in the body. COPREXA has demonstrated the ability to inhibit fibrosis in a number of well established animal models through the sequestration of available copper and inhibition of key fibrotric cytokines, including secreted protein acid rich in cysteine (SPARC), NFkappaB, TGF-beta, FGF-2, IL-1, IL-6, IL-8, and connective tissue growth factor (CTGF).

COPREXA has also completed a one-year, phase I/II clinical trial for the treatment of refractory Idiopathic Pulmonary Fibrosis (IPF), a deadly lung disease. Pending regulatory feedback on such a study, Pipex is planning to launch a phase III registration clinical trial for the treatment of IPF with COPREXA.

About Pipex Pharmaceuticals, Inc.

Pipex Pharmaceuticals, Inc. ("Pipex") is a specialty pharmaceutical company that is developing proprietary, late-stage drug candidates for the treatment of neurologic and fibrotic diseases. Pipex's strategy is to exclusively in-license proprietary, clinical-stage drug candidates and complete the further clinical testing, manufacturing and regulatory requirements sufficient to seek marketing approval in the U.S. and Europe. Pipex is developing products for the treatment of neurologic Wilson's Disease, Idiopathic Pulmonary Fibrosis (IPF), Alzheimer's Disease, Multiple Sclerosis, Dry Age Related Macular Degeneration (AMD), and Fibromyalgia. For further information, please visit

For further information, please visit This press release contains forward-looking statements, within the meaning of Section 21E of the Securities Exchange Act of 1934, that reflect Pipex Pharmaceuticals, Inc. and subsidiaries ("we" or "our") current expectations about its future results, performance, prospects and opportunities, including statements regarding the potential use of COPREXA for the treatment of Huntington's disease, Alzheimer's disease, inflammatory and fibrotic diseases, as well as the prospects for regulatory filings in the treatment of neurologic Wilson's disease, including the filing of an NDA with the FDA during November 2007 and that such NDA will be accepted for filing by the FDA and/or that the FDA will agree with our analysis of data supporting the safety, clinical efficacy, manufacturing, stability and other regulatory requirements necessary for COPREXA to be approved for use in neurologically-presenting Wilson's disease or that, even if it is approved for this initial indication, that we will be able to conduct and complete necessary initial and registration clinical trials required to support and receive FDA approval for a Supplemental New Drug Application to market COPREXA for the treatment of Huntington's disease or other disease indications such as idiopathic pulmonary fibrosis, for example. Where possible, the Company has tried to identify these forward-looking statements by using words such as "anticipates," "believes," "intends," or similar expressions. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements, including risks set forth in our filings with the Securities and Exchange Commission. We cannot assure you that we will be able to successfully develop or commercialize products based on our technologies, including COPREXA™, TRIMESTA™, Zincmonocysteine, CORRECTA, SOLOVAX™, EFFIRMA™ or Anti-CD4 802-2, particularly in light of the significant uncertainty inherent in developing, manufacturing and conducting preclinical and clinical trials of new pharmaceuticals and obtaining regulatory approvals, that our technologies will prove to be safe and effective, that our cash expenditures will not exceed projected levels, that we will be able to obtain future financing or funds when needed, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that we will be able to successfully obtain any further grants and awardsor maintain our existing grants which are subject to performance, that we will be able to patent, register or protect our technology from challenge and products from competition or maintain or expand our license agreements with our current licensors, or that our business strategy will be successful. All forward-looking statements made in this press release are made as of the date hereof, and the Company assumes no obligation to update the forward-looking statements included in this news release whether as a result of new information, future events, or otherwise, other than as required by law.

Contact Information

  • For Further Information Contact:

    Steve H. Kanzer, CPA, Esq.
    Chairman and Chief Executive Officer
    (734) 332-7800

    Thomas Redington, Ph.D. (investor relations)
    Redington, Inc.
    (203) 222-7399